With approximately 3.5 million Americans living with Autism Spectrum Disorder (ASD) and each person spending roughly $2.4 million over their lifespan, there’s a desperate call for a cure or treatment.(1) As such, Robert Naviaux may have found some answers with his new research findings that suggest autism symptoms can be reversed.

On average, 1 in 68 children have ASD according to The Centers for Disease Control and Prevention. Based on these statistics, this condition is four times more common in boys than girls. (2)

Currently, there is no single approved drug that targets the core symptoms of autism according to Dr. Wendy Roberts, medical director at Integrated Services for Autism and Neurodevelopmental Disorders. She said, “We tend to borrow drugs from disorders like anxiety or inattention, so if we could find a drug that is helpful for a significant number of children and young people with autism, that would be great.” (3)

A professor of medicine, pediatrics, and pathology at US San Diego School of Medicine may have scratched the surface about a potential drug that can help children with ASD.

Robert Naviaux’s Research on Suramin and ASD

Robert Naviaux, MD, Ph.D., co-director of the Mitochondrial and Metabolic Disease Center at UC San Diego School of Medicine, together with his colleagues conducted a clinical study in 2017 that tested a 100-year old drug called suramin in children with ASD.

This clinical trial began with a double-blind, placebo-controlled safety study that involved 10 boys, aged 5 to 14 years old, all diagnosed with ASD. Half of them received a single infusion of suramin, a drug that was initially developed in 1916 to treat sleeping sickness and river blindness caused by parasites. Then, the other five boys received a placebo.

Children with ASD Spoke Their First Sentence After Suramin Infusion

The findings in this research were remarkable. All five boys that received the suramin infusion showed improvements in language and social behavior, restricted and repetitive behaviors and coping skills.

They witnessed a specific milestone in two of the five children that received the suramin infusion. Naviaux said, “We had four non-verbal children in the study, two 6-year-olds and two 14-year-olds. The six-year-old and the 14-year-old who received suramin said the first sentences of their lives about one week after the single suramin infusion. This did not happen in any of the children given the placebo.”

This particular finding was marked by their parents who were asked to record any changes in symptoms during a 6-week CGI only if the symptom lasted for one week.

In addition to their first sentences being spoken, the 14-year old boy showed tremendous improvement after receiving suramin. One of his parents said, “Within an hour after the infusion, he started to make more eye contact with the doctor and nurses in the room. There was a new calmness at times, but also more emotion at other times. He started to show an interest in playing hide-and-seek with his 16-year-old brother. Nothing has come close to all the changes in language and social interaction and new interests that we saw after suramin. We saw our son advance almost three years in development in just six weeks.”

But the benefits were temporary. The improvements they witnessed in the boy’s cognitive functions, and behavior peaked, and then they slowly faded when the single dose of suramin wore off. But for its initial testing, it gave notable results that it worked on some level.

What is Autism Spectrum Disorder (ASD)?

Autism Spectrum Disorder is characterized by a range of developmental disorders such as communication and language difficulties, repetitive behaviors and inability to socialize. It begins in childhood and continues into adolescence and adulthood with symptoms appearing in the first 5 years of life. (4)

The level of intellectual functionality is extremely variable, extending from profound impairment to superior levels. ASD may be accompanied by other conditions such as epilepsy, depression, anxiety and attention deficit hyperactivity disorder (ADHD). (5)

Furthermore, the prevalence of autism in U.S. Children has dramatically grown since 2000 from 1 in 150 children to 1 in 68 children. According to the World Health Organization (WHO), the possible explanation for the growing prevalence rate is improved awareness, expansion of diagnostic criteria, better diagnostic tools and improved reporting. (6)

What Does This Mean for Families Affected by Autism?

Naviaux’s research was done on a small sample basis, and thus, it needs to be tested on a larger scale with more diverse cohorts of people with ASD that can further confirm these initial findings.

Naviaux stresses that the design and results of the study can only be concluded with the possibilities that their results are wrong because it was done on an extremely small scale or maybe he says, “this opens a renaissance in drug development – even a drug that works like suramin could offer some hope for improving the future of the lives of many kids.”

It’s important to note that suramin is not an approved treatment for autism. It isn’t commercially sold, and it poses serious threats when administered improperly at the wrong dose and schedule, and without monitoring for toxicity and drug levels.

How Long Until Doctors and Scientists Know the True Impact of This Discovery?

These findings are relatively recent, and according to Naviaux, this particular study was expensive. He said, “we did not have enough funding to do a larger study. And even with the funding, we were able to raise; we had to go $500,000 in debt to complete the trial.”

But they’re optimistic that the initial findings will lead to further studies. He says, “we have plans for five additional studies over the next five years to collect all the data the FDA will need to decide about the approval of suramin for autism.” In the meantime, they strongly caution against the unauthorized use of suramin.